Gemtuzumab ozogamicin for relapsed or primary refractory acute myeloid leukemia in children—the Polish Pediatric Leukemia and Lymphoma Study Group experience

• DOI: 10.3389/fimmu.2023.1268993
• Publication Date: 2023-12-22T04:24:52Z
• Authors: Katarzyna Pawinsk...; Malgorzata Czogala; Szymon Skoczen; Marta Surman; Monika Rygielska; Teofila Ksiazek; Agnieszka Pac; Aleksandra Wieczorek; Jolanta Skalska-S...; Magdalena Samborska; Jacek Wachowiak; Radoslaw Chaber; Renata Tomaszewska; Tomasz Szczepanski; Karolina Zielezinska; Tomasz Urasinski; Malgorzata Moj-Ha...; Krzysztof Kalwak; Marta Kozlowska; Ninela Irga-Jaworska; Walentyna Balwierz; Karolina Bukowska...
• Keywords: Gemtuzumab ozogamicin; Calicheamicin; Refractory (planetary science)

Background Gemtuzumab ozogamicin (GO), one of the first targeted drugs used in oncology, consists an anti-cluster differentiation 33 (CD33) monoclonal antibody bound to a derivative cytotoxic calicheamicin. After drug withdrawn 2010 due significantly higher rate early deaths, GO regained approval 2017 for treatment newly diagnosed, refractory, or relapsed acute myeloid leukemia (AML) adults and children over 15 years age. The objective study was retrospective analysis clinical characteristics, outcomes, toxicity profile with primary refractory (R/R) AML treated Poland from 2008 2022. Methods Data were collected through Polish Registry Acute Myeloid Leukemia. From January December 2022, 35 R/R seven centers Pediatric Leukemia Lymphoma Study Group. Results Most (30 35) received only cycle combination various chemotherapy cycles (IDA-FLA, DOXO-FLA, FLA, FLAG, others). Eighteen (51%) achieved complete remission (CR), 14 did not respond treatment, three progressed. therapy followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) 18 CR. 5-year overall survival (OS) after 37.1% ± 8.7% total cohort. There trend toward superior outcome patients strong expression CD33 (over 50% positive cells) compared that lower (OS, 41.2% 11.9% versus 27.8% 13.2%; p = 0.5; event-free survival, 35.4% 11.6% 25.7% 12.3%; 0.5, respectively). Children under have better 34.9% 10.4% 30% 14.5%, 0.3). most common adverse events bone marrow aplasia, fever unknown origin, infections, elevated liver enzyme elevation. Sinusoidal obstruction syndrome occurred two children. Conclusions use severely pretreated children, including those age, previous failure is feasible effective bridging allo-HSCT acceptable profile.